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Table 3 Scenarios for Example 2

From: Flexible Two-Phase studies for rare exposures: Feasibility, planning and efficiency issues of a new variant

Variables and parameters required for set-up

Formulas and values of parameters

Stratification/Proxy Z (with J strata)

Environmental exposure E and Gene proxy SG

 

J = 4

 

Z = 1: E- SG -, Z = 2: E- SG +, Z = 3: E+ SG -, Z = 4: E+ SG +

Phase One prevalence among controls (Ï„0 j):

Ï„0 1 = Pr(E-)Pr(SG -) = (1 - PE)[(1-Se)PG+Sp(1-PG)]

PE = 20%

Ï„0 2 = Pr(E-)Pr(SG +) = (1 - PE)[SePG+(1-Sp).(1-PG)]

PG = 1%

Ï„0 3 = Pr(E+)Pr(SG -) = PE[(1-Se)PG+Sp(1-PG)]

 

Ï„0 4 = Pr(E+)Pr(SG +) = PE[SePG+(1-Sp).(1-PG)]

Risk factor X (with K outcomes)

Exposure to E and exposure to G: K = 4

 

X = 1: E- G-, X = 2: E- G+, X = 3: E+ G-, X = 4: E+ G+

Disease Model (Odds Ratios ψk)

ψ1 = 1, ψ2 = 3, ψ3 = 2, ψ4 = ψ2 × ψ3 × ORI = 30

Phase Two prevalence of X among controls by stratum (Ï€0 jk)

Z = 1: π0 11 = (1 -PE)Sp(1-PG)/Pr(SG -),

 

π0 12 = 1 - π0 11, π0 13 = π0 14 = 0

 

Z = 2: π0 21 = (1 - PE)(1 - Sp)(1-PG)/Pr(SG +),

 

π0 22 = 1 - π0 21, π0 23 = π0 24 = 0

 

Z = 3: π0 31 = π0 32 = 0, π0 33 = PE Sp(1-PG)/Pr(SG -),

 

π0 34 = 1 - π0 33

 

Z = 4: π0 41 = = π0 42 = 0, π0 43 = PE (1 - Sp)(1-PG)/Pr(SG +),

 

π0 44 = 1 - π0 43

  1. *Se = sensitivity; Sp = specificity